Esophageal achalasia is characterized by prolonged occlusion of the lower esophageal sphincter (LES) and reduced peristaltic activity, making it difficult for patients to swallow food and possibly leading to complications such as regurgitation, coughing, choking, aspiration pneumonia, esophagitis, ulceration, and weight loss. Peroral endoscopic myotomy (POEM) is a novel endoscopic procedure that uses the oral cavity as a natural orifice entry point to perform myotomy of the LES. This procedure has the intent of reducing the total number of incisions needed and, thus, reducing the overall invasiveness of surgery.

Background

Estimated U.S. prevalence of achalasia is 10 cases per 100,000, and estimated incidence is 0.6 cases per 100,000 per year. (1) Treatment options for achalasia have traditionally included pharmacotherapy such as injections with botulinum toxin, pneumatic dilation, and laparoscopic Heller myotomy. (1, 2) Although the last two are considered the mainstay of treatment because of higher success rates and relative long-term efficacy compared with pharmacotherapy and botulinum toxin injections, both are associated with a perforation risk of about 1%. Laparoscopic Heller myotomy is the most invasive of the procedures, requiring laparoscopy and surgical dissection of the esophagogastric junction. (2) One-year response rates of 86% and rates of major mucosal tears requiring subsequent intervention of 0.6% have been reported. (3)

POEM is a novel endoscopic procedure developed in Japan by Dr. Haruhiro Inoue et al.(2,4) POEM is performed with the patient under general anesthesia. (5) After tunneling an endoscope down the esophagus toward the esophageal gastric junction, a surgeon performs the myotomy by cutting only the inner, circular LES muscles through a submucosal tunnel created in the proximal esophageal mucosa. POEM differs from laparoscopic surgery, which involves complete division of both circular and longitudinal LES muscle layers. Cutting the dysfunctional muscle fibers that prevent the LES from opening allows food to enter the stomach more easily. (2,5)

Regulatory Status

POEM uses available laparoscopic instrumentation and, as a surgical procedure, is not subject to regulation by the U.S. Food and Drug Administration.

Medical policies are systematically developed guidelines that serve as a resource for Company staff when determining coverage for specific medical procedures, drugs or devices. Coverage for medical services is subject to the limits and conditions of the member benefit plan. Members and their providers should consult the member benefit booklet or contact a customer service representative to determine whether there are any benefit limitations applicable to this service or supply. This medical policy does not apply to Medicare Advantage.

This policy was created in September 2013 and was updated with a search of the MEDLINE database through August 18, 2014. literature on the efficacy of peroral endoscopic myotomy (POEM) primarily comprises small case series and two nonrandomized comparative studies. No randomized controlled trials (RCTs) comparing POEM with other treatment options have been found. Following is a summary of the identified trial and larger series (≥50 patients) on this procedure.

In a nonrandomized trial with historical control, Hungness et al. (2013) reported on perioperative outcomes in patients with achalasia treated with POEM (n=18) or laparoscopic Heller myotomy (LHM) (n=55) at a single U.S. center. (5) Operative times were shorter for POEM than for LHM (113 and 125 minutes, respectively, p<0.05). Additionally, estimated blood loss was less in patients treated with POEM (≤10 mL in all POEM cases vs 50 mL for LHM, p<0.001). Myotomy lengths, complication rates, and length of stay were similar between groups. Pain scores were similar post-anesthesia and post-operatively on the first day, but were higher at 2 hours for POEM patients (3.5 vs. 2.0, p=0.03). Narcotic use was similar between groups, although fewer patients treated with POEM received ketorolac, a nonsteroidal anti-inflammatory drug. POEM patients’ Eckardt scores decreased (median 1 post-operative vs. 7 preoperative, p<0.001), and 16 patients (89%) had treatment success (score≤3) at a median of 6months follow-up. The Eckardt score grades 4 major symptoms of achalasia (dysphagia, regurgitation, retrosternal pain, weight loss) each on a 0 (none) to 3 (severe) scale for a maximum score of 12; total scores of 4 and above represent treatment failure. (6)

In a retrospective study of a prospective database at Oregon Health & Sciences University (Portland, OR), Bhayani et al. (2014) compared outcomes in 37 patients who underwent POEM and 64 patients who underwent LHM for achalasia.7 Full-thickness esophageal injury occurred in 4 POEMs patients, and 8 esophageal and 3 gastric perforations occurred in LHM patients. Mean (SD) hospitalization was 1.1 (0.6) days in the POEM group versus 2.2 (1.9) days in the LHM group (Mann-Whitney U test for all comparisons, p<0.001). Eckardt scores were statistically lower postoperatively in the POEM group compared with the LHM group (p<0.001), but at 6 months (64% of patients assessed), Eckardt scores did not differ statistically between groups (p=0.1). Postoperative decreases in lower esophageal sphincter (LES) pressures were similar between groups. At 6 months, resting LES pressure was higher in the POEM group compared with the LHM group (16 vs 7 mm Hg, p=0.006).

In a prospective case series, von Renteln et al. (2013) reported on 70 patients who underwent POEM at five centers in Europe and North America. (8) mean follow-up period was 10 months (range, 3-12). Follow-up evaluation at 6 months and 1 year showed sustained treatment success of 89% and 82%, respectively. Mean pretreatment Eckardt score was 6.9 compared with 1.3 at 6 months and 1.7 at 1 year (p<0.001 for both comparisons with pretreatment score). In Multivariate analysis, neither age, previous treatment (Botox/dilatation), myotomy length, pre-procedure LES pressure, pretreatment Eckardt score, sex, procedure duration, nor full-thickness dissection during POEM were significant predictors of treatment failure at 1 year. At 3 months after POEM, esophagitis was observed in 42% of cases. However, severity of esophagitis was minor (grade A or B), and all patients could be managed adequately with proton pump inhibitor (PPI) therapy. At 3 months, 22% of patients required occasional and 12% required daily PPI therapy. The 1-year follow-up evaluation showed overall rates of gastroesophageal reflux disease of 37%, and PPI use of 29%. Other complication rates of POEM ranged from 1% to 4%.

The largest published POEM series to date, by Ren et al. (2012), highlighted POEM-specific complications. (13) In their series of 119 cases, 23% of patients developed subcutaneous emphysema intraoperatively and an additional 56%, postoperatively. Three of these patients required treatment with subcutaneous needle decompression. Additionally, 3% patients developed a pneumothorax intraoperatively and another 25% postoperatively. Postoperatively, the incidence of thoracic effusion was 49%, and of mild inflammation or segmental atelectasis of the lungs was 50%. All complications were resolved with conservative treatment.

At least two small case series have evaluated the efficacy and feasibility of POEM for patients with failed Heller myotomy/achalasia recurrence; success rates have been reported in over 90% of cases up to 10 months after rescue POEM. (14,15) Studies also have compared different POEM techniques; comparable outcomes have been reported between patients undergoing full-thickness versus circular myotomy. (16) An international survey of 16 centers (seven in North America, five in Asia, four in Europe, some of which were high-volume centers [≥30 POEMs per center]) reported 841 POEM procedures performed as of July 2012. (17)

Ongoing and Unpublished Clinical Trials

An online search of ClinicalTrials.gov with the search terms “achalasia” and “peroral” and “myotomy” identified 22 active POEM studies. Six registered RCTs are listed in Table 1; none is set in the United States.

Summary of Evidence

Peroral endoscopic myotomy (POEM) is a novel endoscopic procedure for treatment of esophageal achalasia that uses the oral cavity as a natural orifice entry point for lower esophageal sphincter (LES) myotomy. The intent of this approach is to reduce the total number of incisions needed and, thus, the overall invasiveness of surgery. The evidence base comprises case series, cohort studies, and 2 nonrandomized comparative studies. Treatment success at short follow-up periods was reported for a high proportion of patients treated with POEM. However, incidence of adverse events was relatively high, with POEM-specific complications, including subcutaneous emphysema, pneumothorax, and thoracic effusion, reported across studies. Additionally, a substantial proportion of patients undergoing POEM developed esophagitis requiring treatment. In a nonrandomized historical control trial, investigators reported that POEM resulted in shorter operative times and less blood loss than laparoscopic Heller myotomy (LHM), although myotomy lengths, complication rates, length of stay, and narcotic use were similar between surgical groups. A retrospective review showed higher LES pressure at 6 months in patients who underwent POEM compared with those who underwent LHM.

Evidence shows that the POEM technique is evolving and does not yet have a strong evidence base. Uncontrolled case series demonstrated that it can improve symptoms in patients with achalasia, but that adverse effects commonly occur. No studies have determined efficacy and safety compared with a control group, and no comparative effectiveness studies have evaluated long-term outcomes with POEM versus alternative treatment. Therefore, the use of POEM for treatment of esophageal achalasia is considered investigational.

Practice Guidelines and Position Statements

Society of American Gastrointestinal and Endoscopic Surgeons

In 2011, the Society of American Gastrointestinal and Endoscopic Surgeons issued an evidence-based, consensus guideline on the surgical management of esophageal achalasia. The guideline stated that the POEM technique “is in its infancy and further experience is needed before providing recommendations.” (18)

U.S. Preventive Services Task Force Recommendations

Peroral endoscopic myotomy is not a preventive service.

Medicare National Coverage

There is no national coverage determination (NCD). In the absence of an NCD, coverage decisions are left to the discretion of local Medicare carriers.

New Policy. Policy created with literature search through August 1, 2013; considered investigational.

11/20/14

Annual Review. Policy updated with literature review through August 18, 2014; references 3, 6-7, 9-12, and 18 added; no change to policy statement. ICD-9 and ICD-10 diagnosis codes removed; these do not relate to adjudication of this policy.